Clinical Trials Actively Recruiting in the USA

Breakdown for each trial:
• Name of trial
• Short summary
• Countries recruiting
• Link to trial page on clinicaltrials.gov
• Trial Status

A Study of PRN1008 in Adult Patients With Immune Thrombocytopenic Purpura (ITP)

Principia Biopharma is conducting a study of their oral drug PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options and have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. Preclinical findings show that PRN1008 decreases the activity of B cells and other inflammatory-causing immune cells, and in a mouse model of ITP show that PRN1008 can reduce platelet loss.

USA, Australia, Bulgaria, Canada, Czech Republic, Netherlands, Norway, and United Kingdom

https://clinicaltrials.gov/ct2/show/NCT03395210?term=PRN1008&rank=1

Recruiting

A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple- Dose BIVV009 in Participants With Chronic Immune Thrombocytopenia (ITP)

The purpose of this study is to explore the safety, preliminary clinical benefit, and activity of BIVV009 in patients with chronic immune thrombocytopenia. BIVV009 has been designed to block the pathway that triggers the immune system to remove damaged cells. It is believed that this approach may limit the number of cells immune system removes and allow the extra cells to help promote platelet production.

USA

https://clinicaltrials.gov/ct2/show/NCT03275454?recrs=a&cond=ITP&draw=4&rank=30

Recruiting

A study of oral SKI-0-703, SYK inhibitor, in patients with persistent and chronic immune thrombocytopenia

Sponsored by Oscotec Inc., this is a phase 2, multicenter, randomized, double-blind, placebo-controlled, parallel dose study to evaluate the efficacy and safety of SKI-0-703, and SYK inhibitor, in patient’s with persistent and chronic ITP who have failed to respond (or relapsed after trying) other treatments. Patients in this study must have a platelet count less than 30,000μL on two separate occasion as at least 7 days apart. Participants will take part in three treatment groups for twenty weeks.

USA

https://www.clinicaltrials.gov/ct2/show/NCT04056195?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=2

Recruiting

Association of platelet parameters with bleeding severity in children with ITP

Boston Children’s Hospital is conducting an observational cross-sectional study in hopes to replicate larger scale findings from smaller studies that suggest a positive correlation between platelet parameters (such as immature platelet function, absolute immature platelet count, and P-selection and glycoprotein 11b-11a) with bleeding severity in severe immune thrombocytopenia, regardless of platelet counts. This study center hopes to confirm previous findings in a multi-center patient population to investigate the use of these parameters and better predict disease severity and bleeding events.

USA

https://www.clinicaltrials.gov/ct2/show/NCT03810352?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=3

Recruiting

Eltrombopag vs standard front line management for newly diagnosed ITP in children

Through Baylor College of Medicine, this prospective, open-label, randomized two-arm multi-center study focuses on pediatric patients between the ages of 1 years of age – less than 18 years of age, who are newly diagnosed with ITP. Participants will be randomized into groups to receive either eltrombopag or one of three typical first-line ITP therapies. For those in the eltrombopag group, participants can expect to receive this drug for twelve weeks with the option to continue up to one year, depending on their response to the drug. The goal of this study is to determine if platelet counts in pediatric patients with ITP respond better to eltrombopag versus standard first-line therapies.

USA

https://www.clinicaltrials.gov/ct2/show/NCT03939637?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=25

Recruiting

Collection of blood samples from patients with relapsing multiple sclerosis who developed ITP after receiving Lemtrada

This is a global bio-marker project lead by Genzyme-Sanofi that focuses on single nucleotide polymorphism’s (SNPs) from extracted DNA to attempt to better detect which patients will develop drug-induced immune thrombocytopenia after exposure to the drug, Lemtrada, used in the treatment of autoimmune multiple sclerosis. Participants are adults who have developed immune thrombocytopenia following use of Lemtrada for their multiple sclerosis therapy.

USA

https://www.clinicaltrials.gov/ct2/show/NCT03784898?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=38

Recruiting

Safety and tolerability of M254 in healthy volunteers and immune thrombocytopenia patients

This is an open-label two phase study through Momenta Pharmaceuticals, Inc., to look at the safety, tolerability, pharmacodynamics/pharmacokinetics of M254 infusions in healthy patients compared to patients with persistent or chronic immune thrombocytopenia (ITP). M254 is a hypersialylated human IgG with improved tetra-sialylation. The goal of this study is to see if M254 infusions are more effective than standard IVIg infusions alone in the treatment of ITP and other inflammatory disorders. The participants in this study are between the ages of 18-55 years, and can expect to receive either M254 or a placebo if they are healthy, and M254 either alone or in conjunction with IVIg if they have ITP. Administration of IVIg may be at the same time or weeks apart.

USA, Belgium, France, Germany, Hungary, Italy, Netherlands, Poland, Spain

https://www.clinicaltrials.gov/ct2/show/NCT03866577?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=42

Recruiting

A study to assess the ability of Eltrombopag to induce sustained remission in subjects with ITP (TAPER)

This is an open-label, phase 2, prospective single-armed study through Novartis Pharmaceuticals. The goal of this study is to assess the ability of eltrombopag to induce a sustained remission defined as a platelet count at a minimum of 100,000μL in patients with ITP who have failed previous steroid treatment, or relapsed after previous steroid treatment. Participants can expect to receive eltrombopag for two months and then be tapered off. To be eligible, participants with ITP must be adults, with a platelet count under 30,000μL.

USA, Austria, Brazil, France, Chile, Greece, Italy, Japan, Oman, Russian Federation, Spain, Switzerland, Turkey, United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT03524612?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=56

Recruiting

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

This Is a cross sectional multi-site tissue repository for approved investigator-directed biomedical research. Using blood samples and other specimens, researchers from Sanguine Biosciences hope to bridge the gap between researchers and a cure to various diseases, including immune thrombocytopenia. Using DNA collected from various samples, between health and unhealthy controls, the study aims to gain a greater understanding of the etiology of common diseases affecting adults living in the USA.

USA

https://www.clinicaltrials.gov/ct2/show/NCT01931644?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=57

Recruiting

A Phase 2 Study to Evaluate the Safety and Efficacy of KZR-616 in Patients With AIHA ITP (MARINA)

This study led by Kezar Life Sciences is a phase two, randomized, dose-blinded, multicenter study to evaluate the safety and efficacy of KZR-616 (a selective immunoproteasome inhibitor) in the treatment of patients with autoimmune hemolytic anemia (AIHA) and immune thrombocytopenia (ITP). KZR-616 has been shown in earlier analysis to have a positive effect on taming the inflammatory response in other autoimmune conditions in animal models, without causing immunosuppression. Study participants must be over the age of 18 years. For ITP patients specifically, eligibility is restricted to those who have a platelet count under 30,000μL on two separate blood draws over a week apart, or for ITP patients who are receiving on-going treatment and have a platelet count less than 50,000μL.

This study has multiple phases. Other active trials looking at the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of treatment with KZR-616 are now active for the following autoimmune diseases: lupus (MISSION study: https://clinicaltrials.gov/ct2/show/NCT03393013), dermatomyositis and polymyositis (PRESIDIO study: https://clinicaltrials.gov/ct2/show/NCT04033926).

USA

https://clinicaltrials.gov/ct2/show/NCT04039477

Recruiting

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